Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function.
Mucopolysaccharidosis type VII (MPS VII; Sly syndrome) is one of a group of lysosomal storage diseases that share many clinical features, including mental retardation and hearing loss. Lysosomal storage in neurons of the brain and the associated behavioral abnormalities characteristic of a murine mo...
Guardat en:
| Publicat a: | J Clin Invest |
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| Autors principals: | , , , , , , , , |
| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
American Society for Clinical Investigation
1998
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| Matèries: | |
| Accés en línia: | https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC508717/ https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/9525982/ https://ncbi.nlm.nih.govhttps://doi.org/10.1172/JCI1773 |
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